{"id":283743,"date":"2026-02-05T13:02:28","date_gmt":"2026-02-05T18:02:28","guid":{"rendered":"https:\/\/rarediseases.org\/?p=283743"},"modified":"2026-02-05T10:23:48","modified_gmt":"2026-02-05T15:23:48","slug":"nih-director-bhattacharya-to-headline-second-nord-rare-disease-scientific-symposium","status":"publish","type":"post","link":"https:\/\/rarediseases.org\/es\/nih-director-bhattacharya-to-headline-second-nord-rare-disease-scientific-symposium\/","title":{"rendered":"NIH Director Dr. Jay Bhattacharya to Headline Second Annual NORD Rare Disease Scientific Symposium Focused on Accelerating Innovation"},"content":{"rendered":"

Leaders across research, clinical care, industry, and patient advocacy convene to accelerate rare disease breakthroughs from discovery to real-world patient impact<\/em><\/p>\n

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Norwell, Ma., Feb. 5, 2026<\/strong> \u2014<\/strong> The National Organization for Rare Disorders (NORD\u00ae<\/sup>) today announced the agenda for its second annual NORD\u00ae<\/sup> Rare Disease Scientific Symposium<\/a>, taking place April 14\u201315, 2026, in Arlington, Va., and convening leading researchers, clinicians, industry innovators, government leaders, and patient advocates to advance solutions for people living with rare diseases.<\/p>\n

The symposium will feature remarks from NIH Director Dr. Jay Bhattacharya, MD, PhD, underscoring the urgency of accelerating research, diagnosis, and therapeutic development for the more than 30 million Americans living with rare diseases.<\/p>\n

The 2026 Scientific Symposium will feature sessions exploring innovative clinical trial designs, alternates to placebo controls, and emerging regulatory pathways. Attendees will also gain insight into funding models, drug repurposing, and how registries, real-world data, and patient-generated data can inform endpoint selection, evidence generation, and decisions related to access and coverage.<\/p>\n

\u201cWe received incredibly positive feedback from last year\u2019s inaugural symposium,\u201d said Tracey Sikora, NORD Vice President of Research & Clinical Programs. \u201cAttendees consistently cited the sense of camaraderie across multiple rare diseases and the importance of bringing together the community in a shared scientific forum. This year\u2019s program builds on that momentum and is designed to bridge gaps across the rapidly evolving rare disease landscape, particularly in collaborative clinical research, regulatory innovation, and the use of data across the development lifecycle. We\u2019re excited to continue fostering conversations that lead to real solutions.\u201d<\/p>\n

New additions to this year\u2019s program<\/a> focus on regulatory innovation, including drug repurposing and repositioning, rescued therapies, and the U.S. Food and Drug Administration (FDA)\u2019s emerging plausible mechanism pathway.<\/p>\n

Designed as a working forum, the symposium will focus on turning scientific breakthroughs into real-world treatments by fostering collaboration across sectors and strengthening models that move discoveries forward for rare and ultra-rare diseases as well as into scalable solutions benefiting multiple diseases.<\/p>\n

\u201cWe believe in the power of collaboration and connection,\u201d said Pamela Gavin, NORD Chief Executive Officer. \u201cFor more than 40 years, NORD has driven progress in rare disease science. The NORD Rare Disease Scientific Symposium continues this tradition by fostering cross-sector collaboration and helping shape the future of rare disease research and care.\u201d<\/p>\n

Speakers include research and clinical experts from the NORD\u00ae<\/sup> Rare Disease Centers of Excellence<\/a> network, comprised of 46 U.S. leading medical centers and research institutions. Last year\u2019s inaugural event drew more than 600 attendees, underscoring the growing urgency and global interest in advancing rare disease science and accelerating therapeutic development.<\/p>\n

Agenda Highlights<\/strong><\/p>\n